dm+d

Unassigned

New Medicines

LamzedeMetabolic disease - alpha-mannosidosis lysosomal disease

Information

Lamzede
New molecular entity
Chiesi
Chiesi

Development and Regulatory status

Launched
Launched
Phase III Clinical Trials
March 2018
Yes
Yes
Sep 21Lamzede is available as vials containing 10mg powder for solution for infusion. List price for each vial is £886.61 [13].
Sep 21Lamzede has been available in the UK to order since it received its market authorisation on the 23rd of March 2018. However, as it is not nationally reimbursed, needs approval of an individual funding request or must be paid for privately [12].
Jun 20Velmanase alfa for treating alpha-mannosidosis will be scheduled back into the NICE work programme. Timelines for the continuation of this evaluation are to be determined, and are subject to staff capacity and the ongoing management of the COVID-19 situation. Expect this will delay UK launch plans [11].
Jul 19Company has been asked to provide further information to NICE for the HST Committee consideration. A further committee discussion of this HST evaluation has been scheduled to take place on Thursday 29 August 2019 [9].
Feb 19Launched in EU [10].
Oct 18NICE HST evaluation put on hold as Chiesi are currently developing an offering for submission to NHS England (was previously scheduled for November 2019). Assume launch delayed [9].
May 18NICE initial evaluation consultation document indicates that velmanase would not be recommended for NHS funding, due to the high uncertainty in the evidence for benefit and cost effectiveness that would fall well outside the range considered acceptable for highly specialised technologies [8].
Apr 18Approved in EU [7].
Jan 18Positive opinion from the CHMP, indicated for “Enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis.” It is proposed that it be prescribed by appropriately experienced physicians [6].
Oct 16Filed in EU via centralised procedure [5].
Mar 15NCT01681953 (EudraCT Number: 2012-000979-17) completed Feb 15. NCT019087124 follow-on study due to complete Dec 16 [4].
Mar 14No further updates. P3 studies in progress.
Jul 13Granted orphan drug status in the EU in 2005 (EU/3/04/260) [2].

Category

Recombinant human lysosomal alpha-mannosidase
Rare, affects ~460 pts in the EU [2]; 14 pts diagnosed in UK since 1961[NHSC/NIHR]. Deficiency of Laman enzyme results in inability to break down certain carbohydrates, leading to the accumulation of oligosaccharides. Most severe form is infantile phenotype (type 1) leads to rapid progressive mental retardation and typically death between 3 -12 years of age. The juvenile-adult phenotype (type 2) is characterized by a milder, slowly progressive course with survival into adulthood.
Metabolic disease - alpha-mannosidosis lysosomal disease
Intravenous infusion

Further information

Yes

Trial or other data

Apr 20PIII trial in US initiated (NCT04031066) [10].
Aug 13Chiesi is acquiring the company, Zymenex, and the acquisition includes Lamazym [3].
Jul 13NCT01908712: is the Lamazym Aftercare Study FR (rhLAMAN-07)
Jul 13EudraCT Number: 2013-000321-31 A single-centre, uncontrolled, open-labeled PIIIb trial of the long-term safety of Lamazym aftercare treatment of 5 subjects (aged ≥2 years) who previously participated in Lamazym-trials. The primary endpointsare : Adverse events (AEs); vital signs and change in physical examination; and development of Lamazym antibodies and neutralizing/inhibitory antibodies. Start Date: 2013-06-07 [1]
Jul 13EudraCT Number: 2012-000979-17: a multi-center, double-blind, randomized, placebo-controlled, PIII trial, investigating the efficacy and safety of repeated Lamazym treatment of 20 subjects (aged ≥2 years) with alpha-mannosidosis. Primary efficacy endpoints are change from baseline in the active treated group vs the placebo group in the level of oligosaccharides in serum and in the 3-minute stair climb test (3MSCT) assessed at 26 and 52 weeks. Start Date: 2012-12-17 [1].

Evidence based evaluations